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Risk‐adapted autologous stem cell transplantation with adjuvant dexamethasone ± thalidomide for systemic light‐chain amyloidosis: results of a phase II trial
Author(s) -
Cohen Adam D.,
Zhou Ping,
Chou Joanne,
TeruyaFeldstein Julie,
Reich Lilian,
Hassoun Hani,
Levine Beth,
Filippa Daniel A.,
Riedel Elyn,
Kewalramani Tarun,
Stubblefield Michael D.,
Fleisher Martin,
Nimer Stephen,
Comenzo Raymond L.
Publication year - 2007
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2007.06783.x
Subject(s) - medicine , al amyloidosis , thalidomide , multiple myeloma , melphalan , surgery , autologous stem cell transplantation , gastroenterology , dexamethasone , adjuvant , transplantation , adjuvant therapy , cancer , immunology , antibody , immunoglobulin light chain
Summary High‐dose melphalan (MEL) with autologous stem cell transplant (SCT) is an effective therapy for systemic AL amyloidosis (AL), but treatment‐related mortality (TRM) has historically been high. We performed a phase II trial of risk‐adapted SCT followed by adjuvant dexamethasone (dex) and thalidomide (thal) in an attempt to reduce TRM and improve response rates. Patients ( n = 45) with newly diagnosed AL involving ≤2 organ systems were assigned to MEL 100, 140, or 200 mg/m 2 with SCT, based on age, renal function and cardiac involvement. Patients with persistent clonal plasma cell disease 3 months post‐SCT received 9 months of adjuvant thal/dex (or dex if there was a history of deep vein thrombosis or neuropathy). Organ involvement was kidney (67%), heart (24%), liver/GI (22%) and peripheral nervous system (18%), with 31% having two organs involved. TRM was 4·4%. Thirty‐one patients began adjuvant therapy, with 16 (52%) completing 9 months of treatment and 13 (42%) achieving an improvement in haematological response. By intention‐to‐treat, overall haematological response rate was 71% (36% complete response), with 44% having organ responses. With a median follow‐up of 31 months, 2‐year survival was 84% (95% confidence interval: 73%, 94%). Risk‐adapted SCT with adjuvant thal/dex is feasible and results in low TRM and high haematological and organ response rates in AL patients.